Open-label, Safety and Efficacy Evaluation of FX-1006A in Patients With V122I or Wild-type Transthyretin (TTR) Amyloid Cardiomyopathy
This is a Phase 3, open-label study designed to obtain additional, long-term, safety and efficacy data for tafamidis, and to continue to provide subjects who have completed Protocol Fx1B-201 (a Phase 2, open-label study to evaluate TTR stabilization, as well as the safety and tolerability of tafamidis) with 20 mg oral tafamidis (soft gelatin capsule) for up to 10 years or until subject has access to tafamadis for TTR-CM via prescription.
The physical examination (including weight and vital signs) and the relevant end of study clinical laboratory tests (alkaline phosphatase, alanine aminotransferase, aspartate aminotransferase, blood urea nitrogen, gamma glutamyl transferase, creatinine, total bilirubin, international normalized ratio, troponin I, troponin T, and amino-terminal B-type natriuretic peptide) from Protocol Fx1B-201 will be used for Protocol Fx1B-303. If more than 30 days has elapsed between the final study visit of Protocol Fx1B-201 and Day 0 of Protocol Fx1B-303, an abbreviated physical examination (including weight and vital signs) and clinical laboratory assessments must be performed on Day 0.
Eligible patients will begin once-daily dosing with 20 mg Fx-1006A at home on Day 1 (i.e., first dose) and will return to the clinical unit for study visits every 6 months.
Adverse events (AEs) and concomitant medication use will be collected at each 6-month visit to the clinical unit. Blood draws for clinical safety laboratory tests and abbreviated physical examinations (including weight and vital signs) will also be performed at each 6-month clinic visit. ECGs will be performed every 12 months on an annual basis. A telephone call will be made at 3-month intervals between clinic visits to assess safety and use of concomitant medications.
For the evaluation of efficacy, the Patient Global Assessment, NYHA classification, KCCQ, 6-minute walk test, and efficacy-related clinical laboratory tests (serum levels of troponin T, troponin I, and NT-pro-BNP) will be determined every 6 months. In addition, echocardiograms will be performed every 12 months on an annual basis.
An end of study visit including all safety and efficacy assessments will occur upon patient completion of the study, premature withdrawal (for any reason), or in the event of program discontinuation by the Sponsor.
18 Years and up
Accepting Healthy Volunteers?
- Patient successfully completed Protocol Fx1B-201
- If female; patient is post menopausal. If male, female partner is post-menopausal. If female is of child bearing potential, willing to use acceptable method of birth control up to 3 months after last dose (included female partners of male participants)
- Patient is willing to comply with protocol
- Patient did not successfully complete Fx1B-201
- Chronic use of NSAIDS
- Patient has a clinically significant medication condition that increases risk of study participation
- Patient has received heart or liver transplant